I tried to make progress in the following three areas in 2016: 1) Improve Clinical Care, 2) Raise Awareness, and 3) Develop Novel Treatments for Wolfram syndrome. I did my best. I know I could not make a breakthrough, but I will keep on trying.
My highest priority right now is to work out the logistics for the upcoming clinical trial. As our medical center announced in November this year, we plan to start a phase 1b safety clinical trial of dantrolene sodium in patients with Wolfram syndrome. Dantrolene sodium is an FDA-approved drug utilized for the treatment of muscle stiffness. We found that dantrolene could prevent the destruction of insulin-secreting beta cells in animal models of Wolfram syndrome and in brain cells differentiated from skin samples taken from patients with the illness in 2014 after the 12 years of research. This drug can target the molecular pathways altered in Wolfram syndrome in cell and animal models of Wolfram syndrome. Nobody has ever tested dantrolene in patients with Wolfram syndrome, so our first and most important objective is to make sure it’s safe. We will also closely monitor patients’ vision and brain function, as well as the function of their remaining insulin-secreting beta cells based on the data collected through our research clinic study. I sincerely hope that this pilot study will pave the way for the next phase of our work on the development of novel treatments.
I have been working diligently to commence the trial early in 2017, and secure additional funding from the National Institutes of Health to recruit more patients. Please contact our nurse coordinator, Ms. Ashley Simpson (+1-314-286-1550, firstname.lastname@example.org) or check our medical center’s website (http://wolframsyndrome.dom.wustl.edu/clinical-trials/) for more information. I appreciate the support from the Snow Foundation, Ellie White Foundation, Washington University Medical Center, and donations from many people for the upcoming trial.
I have been working with Dr. Tim Barrett in the UK to conduct international clinical trials for Wolfram syndrome. Dr. Barrett’s team will start a phase 2 clinical trial of valproic acid in patients with Wolfram syndrome in 2017. They plan to recruit more than 70 patients in Europe.
In parallel, I will keep on developing novel drugs and regenerative therapies for Wolfram syndrome.
As we near the end of this year, I would like to take this opportunity to thank you all for your continued support. You have provided patients with hope and emotional support. Together with you, we can make a difference in the future of our patients. Thank you again for your generous support and continued encouragement. You gave me the power and courage to continue. I cannot thank you enough. I hope 2017 will be a great year for all of us.